Potential target to prevent leukemia identified

17 November 2014

A team of researchers has identified two proteins that appear to be crucial for the development of acute myeloid leukemia, as well as impacting relapse.

Published in Cell Reports, the study demonstrated that, by targeting these proteins, it was possible to inhibit the development of leukemia. The team used models to show that eliminating certain genes could prevent leukemia from developing further, even when the bone marrow stem cells contained the cancer-causing receptor mutations.

Importantly, the study found that this did not prevent the mice from otherwise producing and maintaining a normal blood system.

The researchers were able to identify several drug compounds that target the proteins - FAK and PAK1 - directly instead of the genes that produce them. They found that these, which have been approved for experimental use in animals but not in humans, that were just as effective in blocking development of leukemia.

The next step is to continue testing and refining those experimental drug compounds to verify their effectiveness for potential testing in human trials, Dr Kapur said.